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Therapeutic Strategies for Spinocerebellar Ataxia Type 1

Spinocerebellar ataxia type 1 (SCA1) is an autosomal dominant neurodegenerative disorder that affects one or two individuals per 100,000. The disease is caused by an extended CAG repeat in exon 8 of the ATXN1 gene and is characterized mostly by a profound loss of cerebellar Purkinje cells, leading t...

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Detalles Bibliográficos
Autores principales: Kerkhof, Laurie M. C., van de Warrenburg, Bart P. C., van Roon-Mom, Willeke M. C., Buijsen, Ronald A. M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10216181/
https://www.ncbi.nlm.nih.gov/pubmed/37238658
http://dx.doi.org/10.3390/biom13050788